Gene therapy is revived

Various medical and ethical problems have meant that its use has stalled for some years

There is no doubt that gene therapy has been a major medical breakthrough, but various medical and ethical problems have meant that its use has stalled for some years now.

This treatment was first used in 1990, when a four-year-old girl with a genetic disease, adenosine deaminase (ADA) deficiency was treated with gene therapy. The child initially improved, but the change was temporary and she eventually had to continue standard treatment. Nevertheless, the experiment was shown to be safe and moderately effective, thus encouraging the launch of other similar clinical trials.

However, in 1999, a young 18-year-old man died after undergoing gene therapy. Later, in 2002, another young 18-year-old man with severe immune deficiency who underwent gene therapy in France developed leukaemia. Two other young people who participated in the same trial suffered the same problem, and another patient in the United Kingdom also developed leukaemia. As a result, the health authorities in France, Germany, the United Kingdom and the United States halted all clinical trials in which gene therapy was used. We must not forget, however, that the French trial also had positive outcomes, as nine of the ten young people included improved considerably.

Despite the prohibition, the research continued, and safer and more effective techniques were developed, so that little by little, the use of gene therapy has been rekindled.

Several institucions have now set up new gene therapy trials

Several universities and commercial companies have now set up new gene therapy trials to treat various diseases.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.